Clinical trials: Congenital Myopathy
Healthy Volunteer, Becker Muscular Dystrophy
A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adults
EDG-5506 is an investigational product intended to protect and improve function of dystrophic muscle fibers. This Phase 1 study of EDG-5506 will assess the safety, tolerability, and pharmacokinetics (PK) and of EDG-5506 in adult…
- Trial name
- NCT04585464
- Testing
- EDG-5506
- Where
- United States
- Volunteers
- 127 people, healthy volunteers welcome
- Sponsor
- Edgewise Therapeutics, Inc.
Behcet's Disease, Fibromyalgia
Evaluation of Fibromyalgia With Disease Activity and Clinical Findings in Women With Behçet's Disease
In the literature, the relationship between fibromyalgia and disease activity has been assessed in a few studies without discrimination between women and men.In this study, it was aimed to evaluate the relationship between…
- Trial name
- NCT03543709
- Testing
- fibromyalgia 2013 criteria, Behcet's disease current activity form
- Where
- Turkey (Türkiye)
- Volunteers
- 72 people
- Sponsor
- Bursa Yuksek Ihtisas Training and Research Hospital
Myotonic Dystrophy
The United Kingdom National Registry for Myotonic Dystrophy
Myotonic dystrophy (dystrophia myotonica - DM) exists in two forms, usually referred to as DM1 (type 1) and DM2 (type 2). Both conditions are genetic disorders but each affects a different gene.
- Trial name
- NCT04003363
- Testing
- Patient Registry
- Where
- United Kingdom
- Volunteers
- 900 people
- Sponsor
- Newcastle University
Duchenne Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy
Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next Study
The objective of the ActiLiège Next study is to collect longitudinal data from patients and control subjects using a wearable magneto-inertial device.
- Conditions
- Duchenne Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy 1, Charcot-Marie-Tooth
- Trial name
- NCT05982119
- Testing
- ActiMyo/Syde
- Where
- 8 sites in Belgium, Czechia and 5 more
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Muscular Dystrophies
A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
The purpose of this study is to determine whether GSK2402968 is effective in the treatment of ambulant boys with Duchenne muscular dystrophy resulting from a mutation thought to be corrected by exon 51 skipping.
- Trial name
- DMD114044 (NCT01254019)
- Testing
- GSK2402968 6mg/kg/week
- Where
- 47 sites in Argentina, Belgium and 18 more
- Volunteers
- 186 people
- Sponsor
- GlaxoSmithKline
Multiple Sclerosis, Spinal Cord Injury
Power Over Pain (POP) Study
Many individuals with multiple sclerosis (MS), spinal cord injury (SCI), acquired amputation (AMP), muscular dystrophy (MD), and low back pain (LBP) experience pain. There has been little research on how to treat this pain.
- Conditions
- Multiple Sclerosis, Spinal Cord Injury, Chronic Pain, Other and Unspecified Amputation Stump Complications
- Trial name
- POP (NCT01800604)
- Testing
- Pain Self-Management Intervention #1, Pain Self-Management Intervention #2, Pain Self-Management Intervention #3
- Where
- United States
- Volunteers
- 177 people
- Sponsor
- University of Washington
Muscular Dystrophy, Duchenne
Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy
This study seeks to develop and validate non-invasive assessments of cardiac and respiratory muscles with magnetic resonance imaging (MRI) to better predict the natural disease progression of Duchenne muscular dystrophy (DMD) in…
- Trial name
- CPI (NCT02195999)
- Testing
- Magnetic Resonance Imaging (MRI), Pulmonary Function Testing (PFT), Metabolic Exercise Testing using stationary bicycle
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- University of Florida
FSH Muscular Dystrophy, Muscular Dystrophy
Amino Acids and Exercise in FSHD
This study evaluates whether amino acid supplementation, combined with a structured diet and exercise program, improves body composition (increased muscle mass and reduced fat mass) and physical performance in individuals with…
- Trial name
- NCT07543016
- Testing
- Essential Amino Acids Supplementation intervention, Exercise, Diet
- Where
- Italy
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- University of Pavia
Muscular Dystrophy, Duchenne
A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of 5 escalating doses of SRP-5051 (vesleteplirsen) administered as a single dose to patients with DMD amenable to exon 51 skipping…
- Trial name
- NCT03375255
- Testing
- SRP-5051
- Where
- 8 sites in United States, Canada
- Volunteers
- 15 people
- Sponsor
- Sarepta Therapeutics, Inc.
Myotonic Dystrophy 1, DM1
The Spanish National Registry for Myotonic Dystrophy Type 1
Myotonic Dystrophy Type 1 (DM1) is a rare genetic neuromuscular condition that can affect multiple organs and varies widely in how it presents.
- Conditions
- Myotonic Dystrophy 1, DM1, Myotonic Dystrophy Type 1, Myotonic Dystrophy, Congenital
- Trial name
- DM1-Hub (NCT07385443)
- Testing
- Patient Registry
- Where
- 8 sites in Spain
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Fundació Institut Germans Trias i Pujol
Duchenne Muscular Dystrophy
Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients
The specific aim of this Phase I/II study is to assess the safety of intravenous administered Morpholino oligomer directed against exon 51 (AVI-4658 PMO).
- Trial name
- NCT00844597
- Testing
- AVI-4658 for Injection
- Where
- 2 sites in United Kingdom
- Volunteers
- 19 people
- Sponsor
- Sarepta Therapeutics, Inc.
Non Invasive Prenatal Diagnosis
NIPD on CFTC for Triplet Repeat Diseases
The purpose of this study is to develop and validate an analytical and clinical NIPD test for triplet repeat diseases by isolated circulating fetal trophoblastic cells (CFTC) analysis from maternal blood, searching for the…
- Trial name
- DIACCIMEX (NCT03087526)
- Testing
- Non invasive prenatal diagnosis
- Where
- 8 sites in France
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University Hospital, Montpellier
Muscular Dystrophy, Duchenne
Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy
The primary objective of this study is to explore safety and tolerability of eteplirsen in participants with advanced stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
- Trial name
- NCT02286947
- Testing
- Eteplirsen
- Where
- 9 sites in United States
- Volunteers
- 24 people
- Sponsor
- Sarepta Therapeutics, Inc.
Inclusion Body Myositis, Myotonic Dystrophy 1
The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.
The planned project is an intervention study to assess the risk of falling after adaptation of an assistive gait devices in patients with the following neuromuscular diseases: Inclusion body myositis, myotonic dystrophy, limb…
- Conditions
- Inclusion Body Myositis, Myotonic Dystrophy 1, Myotonic Dystrophy 2, Facio-Scapulo-Humeral Dystrophy
- Trial name
- Risk of fall (NCT07072676)
- Testing
- Assistive gait devices combined with physiotherapy
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- LMU Klinikum
Muscular Dystrophies, Muscular Dystrophy
Muscle Tissue Bank for Muscular Dystrophy
This protocol proposes to establish gene expression profiles of muscular dystrophies for correct diagnosis and for development of experimental therapies for these diseases.
- Trial name
- NCT01950897
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- Wake Forest University Health Sciences
Duchenne Muscular Dystrophy
Open-label Extension of the HOPE-2 Trial
This Phase 2, multi-center, open-label extension trial will provide deramiocel (CAP-1002) to subjects that were enrolled in the HOPE-2 trial and completed 12 months of follow-up.
- Trial name
- HOPE-2-OLE (NCT04428476)
- Testing
- Deramiocel (CAP-1002)
- Where
- 5 sites in United States
- Volunteers
- 13 people
- Sponsor
- Capricor Inc.
Facioscapulohumeral Muscular Dystrophy
Rasch-analysis of Clinical Severity in FSHD
The purpose of this study is to make a standardized and scalable Rasch-built clinical severity scale to help in finding genetic and environmental modifiers of disease in Facioscapulohumeral muscular dystrophy (FSHD).
- Trial name
- ROC-FSHD (NCT02766985)
- Where
- 2 sites in United States
- Volunteers
- 44 people
- Sponsor
- University of Kansas Medical Center
Myotonic Dystrophy Type 1, Congenital DM1
Remote Assessments and Genetic Determinants of Congenital and Childhood Myotonic Dystrophy
Myotonic dystrophy type 1 (DM1) can affect people in many different ways, even in the same family. The symptoms that children experience can be different and more severe than adults.
- Conditions
- Myotonic Dystrophy Type 1, Congenital DM1, Juvenile DM1, Childhood DM1
- Trial name
- REACH-DM KIDS (NCT07630389)
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- University of Rochester
Duchenne Muscular Dystrophy
Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study
This is an open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD (Duchenne's muscular dystrophy) patients who have been previously treated in one of the GIVINOSTAT studies.
- Trial name
- NCT03373968
- Testing
- Givinostat
- Where
- 39 sites in United States, Belgium and 9 more
- Volunteers
- 206 people
- Sponsor
- Italfarmaco
Duchenne Muscular Dystrophy
Extracellular RNA Biomarkers of Duchenne Muscular Dystrophy
Current methods of measuring the response to new treatments for muscular dystrophies involve the examination of small pieces of muscle tissue called biopsies.
- Trial name
- NCT05016908
- Where
- 2 sites in United States
- Volunteers
- 100 people
- Sponsor
- Massachusetts General Hospital
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